An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
Study Details
Study Description
Brief Summary
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome.
Condition or Disease | Intervention/Treatment | Phase |
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Phase 2 |
Detailed Description
The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution in children and adolescents with Prader-Willi Syndrome. The secondary purpose is to investigate measures of efficacy. Subjects will receive treatment with NNZ-2591 oral solution (50 mg/mL) doses for a total of 13 weeks.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: NNZ-2591 NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks. |
Drug: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Other Names:
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Outcome Measures
Primary Outcome Measures
- Safety and Tolerability [13 weeks]
To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.
- Pharmacokinetic - Measurement of Cmax [13 weeks]
Maximum observed concentration (Cmax) of NNZ-2591
- Pharmacokinetic - Measurement of AUC [13 weeks]
Area under the concentration-time curve of NNZ-2591
- Pharmacokinetic - Measurement of time to Cmax [13 weeks]
Time to Cmax of NNZ-2591
- Pharmacokinetic - Measurement of t1/2 [13 weeks]
Apparent terminal elimination half-life of NNZ-2591
Secondary Outcome Measures
- Exploratory efficacy measurement [13 weeks]
Assessed by PWS-specific Clinical Global Impression Scale & Domain -Overall Improvement Score (CGI-I)
- Exploratory efficacy measurement [13 weeks]
Assessed by Caregiver Global Impression-Change Score
- Exploratory efficacy measurement [13 weeks]
Assessed by PWS-specific Clinical Global Impression Scale-Severity (CGI-S) Overall Score
- Exploratory efficacy measurement [13 weeks]
Assessed by Caregiver Top 3 Concerns Likert Scale Scores
- Exploratory efficacy measurement [13 weeks]
Assessed by PWS Profile Score
- Exploratory efficacy measurement [13 weeks]
Assessed by PWS Anxiousness and Distress Behaviors Questionnaire Score (PADQ)
- Exploratory efficacy measurement [13 weeks]
Assessed by Autism Diagnostic Observation Schedule (ADOS-2), Repetitive behaviors and Social scores
- Exploratory efficacy measurement [13 weeks]
Assessed by Hyperphagia Questionnaire-Clinical Trials (HQ-CT) Score
- Exploratory efficacy measurement [13 weeks]
Assessed by Food Safety Zone Questionnaire Score
- Exploratory efficacy measurement [13 weeks]
Assessed by Vineland Adaptive Behavior Scales-3 Growth Scale Scores
- Exploratory efficacy measurement [13 weeks]
Assessed by Zarit Burden Interview Score
- Exploratory efficacy measurement [13 weeks]
Assessed by Child Sleep Habits Questionnaire (CSHQ)
- Exploratory efficacy measurement [13 weeks]
Assessed by Impact of Childhood Neurological Disability Scale (ICND)
- Exploratory efficacy measurement [13 weeks]
Assessed by Quality of Life Inventory-Disability (QI-Disability)
- Exploratory efficacy measurement [13 weeks]
Assessed by Kaufman Brief Intelligence Test or Mullen Scales of Early Learning
- Exploratory efficacy measurement [13 weeks]
Assessed by PWS Suicidality Assessment
- Exploratory efficacy measurement [13 weeks]
Assessed by Caregiver Diary
Eligibility Criteria
Criteria
Inclusion Criteria:
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Clinical diagnosis of PWS with a documented disease-causing genetic abnormality of the chromosome 15q11-q13 confirmed by DNA methylation and microarray.
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Males or females aged 4-12 years, inclusive.
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Body weight of 12 kg to 100kg (inclusive) at Baseline.
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Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit.
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Must currently be on treatment with growth hormone.
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Each subject must be able to swallow the study medication provided as a liquid solution.
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Caregiver(s) must have sufficient English language skills.
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Subject and caregiver must reside in the US and have been resident in the US for at least 3 months prior to screening.
Exclusion Criteria:
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Body weight <12 kg or >100 kg at Baseline.
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HbA1c values above 7% at the Screening visit.
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Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
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Positive pregnancy test at the Screening visit.
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Positive drugs of abuse screen not explained by concomitant medications.
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Abnormal QTcF interval or prolongation at Screening.
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Any other clinically significant finding on ECG at the Screening visit.
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Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or Baseline.
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Previous COVID 19 infection with last 12 months that required hospitalization.
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Previous COVD-19 infection involving multi-organ systems, resulting in Multisystem Inflammatory Syndrome in Children (MIS-C) or with clinically significant long term effects.
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COVID-19 infection associated with acute kidney injury (AKI) or renal conditions.
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Renal conditions or abnormalities identified in laboratory testing, imaging or medical history.
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Liver conditions and Hepatic abnormalities.
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Vision abnormalities and Ocular conditions.
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Excluded concomitant treatments.
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Unstable seizure profile.
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Current clinically significant cardiovascular, gastrointestinal, or respiratory disease, or clinically significant organ impairment, or endocrine disease with the exception of obesity and controlled hypothyroidism.
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Current clinically significant hypo or hyperthyroidism, Type 1 or Type 2 diabetes mellitus requiring insulin (whether well controlled or uncontrolled), or uncontrolled Type 1 or Type 2 diabetes.
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Has planned surgery during the study.
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History of, or current, cerebrovascular disease or brain trauma.
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History of, or current catatonia or catatonia-like symptoms.
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History of, or current, malignancy.
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Current major or persistent depressive disorder (including bipolar depression).
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Significant uncorrected hearing impairment.
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Allergy to strawberry.
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Has participated in another interventional clinical study within 30 days prior to start of Screening.
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Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Neuren Pharmaceuticals Limited
Investigators
- Study Director: James Shaw, Neuren Pharmaceuticals
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- NEU-2591-PWS-001